FIXX - Homology Medicines unveils gene therapy program targeting MPS II

Homology Medicines ([[FIXX]] +3.5%) has announced a new in vivo gene therapy development program for the treatment of mucopolysaccharidosis type II (MPS II), or Hunter syndrome.Development candidate HMI-203 is a potential one-time AAVHSC treatment designed to deliver functional copies of the IDS gene to multiple target organs following a single I.V. administration. The company has initiated pivotal IND-enabling studies.MPS II is a rare, X-linked lysosomal storage disorder caused by mutations in the iduronate-2-sulfatase ((IDS)) gene, which is responsible for producing the I2S enzyme that breaks down large sugar molecules, or cellular waste, called glycosaminoglycans (GAGs). Accumulation of GAGs causes progressive debilitation and decline in intellectual function.

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Homology Medicines unveils gene therapy program targeting MPS II